Revisiting the management of chronic ITP; a randomized controlled clinical trial.

The balance between therapy effectiveness and economic efficiency in chronic immune thrombocytopenia (ITP) becomes more confusing. This single-center open label randomized controlled trial evaluates the effectiveness and safety of hydroxychloroquine in chronic ITP patients against other affordable second-line treatments. It is registered under number (NCT03229746) at Clinical Trials.gov. 120 patients were recruited and randomly allocated to three arms of hydroxychloroquine, vincristine, and azathioprine equally. Platelet counts of more than 100 × 109/L were interpreted as complete response (CR), while response (R) was determined as platelet counts ranging from 30 × 109/L to less than 100 × 109/L with the doubling of the pretreatment platelet count. Overall response (OR) was defined to include both CR and R. Patients were monitored every 6 weeks for a total of 24 weeks. The population baseline characteristics regarding age, sex, duration of the disease, baseline platelets count, and presence of antinuclear antibodies ANA or antiplatelet antibodies were similar among tested groups. There was a significant difference in the overall response between hydroxychloroquine (80.6%) and azathioprine (55.9%) (p-value <0.05). This difference was not significant between hydroxychloroquine and vincristine group (63.2%) (p-value = 0.09). This study proves that hydroxychloroquine can contribute to the therapy of chronic ITP especially as an affordable and well-tolerated drug.

Monocytic myeloid-derived suppressor cells in chronic lymphocytic leukemia patients: a single center experience.

This study aimed to analyze the frequency of peripheral Mo-myeloid-derived suppressor cells (Mo-MDSCs) in newly diagnosed CLL patients and to correlate their level with other prognostic factors such as frequency of CD38 cells and ZAP-70 cells and with the clinical response and survival outcomes in these patients. Fifty CLL patients and 20 age-matched healthy controls were included in this study. Flow cytometric detection of ZAP 70, CD38, and Mo-MDSCs was done. Mo-MDSC levels wer significantly higher in CLL patients (27.51 ± 1.70) than healthy controls (16.79 ± 0.66; p < .0001). Higher levels of Mo-MDSCs were detected in advanced Rai clinical staging than Stage I. Mo-MDSCs level was significantly correlated with the frequency of CD38 (r = 0.505; p < .0001) and ZAP-70 cells (r = 0.421; p < .0001). Higher levels of Mo-MDSCs predict poor survival in CLL patients with Mo-MDSCs levels <25% (n = 21) versus >25% (n = 29; log - Rank test, p < .0001). In conclusion, Mo-MDSCs are correlated with tumor progression and a poor prognosis in CLL.

More about the combination of rituximab, cyclosporine and dexamethasone in the treatment of chronic ITP. A useful option on an environment with limited resources.

Treatment of chronic primary immune thrombocytopenia (ITP) is challenging especially with limited resources and therapy-related complications. This prospective interventional study assessed the efficacy and safety of triple therapy(TT4); a combination treatment of oral dexamethasone 40 mg for days1-4, oral cyclosporine A 2-3 mg/kg daily for 28 consecutive days and intravenous rituximab 100 mg for days7,14,21 and 28 among 40 patients with ITP who failed previous 2 or more treatment options. Our aim was to maintain platelet count ≥ 30 X 109/L without any obvious bleeding at any point in the study. Platelet counts were assessed weekly for a month then monthly for 2 years to evaluate the long-term response. TT4 was well tolerated and induced a good response with a significant increase in the mean platelet count after the 1st, 2nd, 3rd, and 4thweek compared to the baseline. Patients with mean platelet count ≥ 30 X 109/L at the 6th month were 75% (30/40 patients). Treatment free survivals (TFS) at 12 and 24 months were 93.3% (28/30 patients) and 80% (24/30 patients) respectively. TT4 is an effective treatment option that maintained platelet count in the desired level and induced a higher sustained response, especially in an environment with limited resources.

Lower Transcranial Doppler Flow Velocities in Sickle Cell Anemia Patients on Hydroxyurea: Myth or Fact.

Transcranial Doppler (TCD) detects stroke risk in patients with sickle cell anemia (SCA). Hydroxyurea therapy has the ability to induce increased levels of fetal hemoglobin in sickle cells thus decreasing tendency for red cell sickling. This study aimed to evaluate TCD findings in SCA patients on hydroxyurea and correlate the time-averaged mean velocity (TAMV) with their hematological parameters. Forty SCA patients of both sexes, aged 16-22 years with no history of stroke were screened with TCD for an elevated TAMV, divided into: Group T (20 patients on blood transfusion); and Group H (20 patients on daily hydroxyurea). For all, full medical history, clinical examination, hemoglobin, hematocrit, leukocytes, platelets, fetal hemoglobin and sickling test, in addition TCD to describe the pattern of cerebral blood flow abnormalities were done. TAMV in all cerebral arteries were significantly higher in Group T than Group H, the highest TAMV (147.5 ± 57.09 cm/s) was found in the right middle cerebral artery and correlated negatively with hematocrit in Groups H (P < 0.001). There were 2 (10%) abnormal TAMV results and 5 (25%) conditional in Group T, while all results were normal in Group H. Hydroxyurea therapy may lower TCD velocities and prevent the risk of primary stroke in SCA patients.

Could intradialytic nutrition improve refractory anaemia in patients undergoing haemodialysis?

BACKGROUND: This prospective randomised study was designed to evaluate the efficacy of intradialytic parenteral nutrition (IDPN) therapy in malnourished patients with refractory anaemia. METHODS: Forty patients who were malnourished with a BMI not greater than 23 (17-23) kg/m2 , undergoing regular HD were included. Of those, 20 patients received 500-1000 ml of IDPN at a rate of 250-300 ml/h at each HD session three days per week for six consecutive months. The other 20 patients did not receive IDPN infusion. The malnutrition inflammation score (MIS) and haematological parameters were recorded at baseline and after three and six months. RESULTS: Mean haemoglobin levels, BMI and serum albumin were significantly increased while MIS was significantly decreased after the 3rd and 6th months of IDPN. CONCLUSION: IDPN has a good role in improving refractory anaemia by significantly increasing haemoglobin levels, body weight, and serum albumin levels. The intervention also significantly decreases the MIS of patients.

Evaluation of pulmonary function changes in children with type 1 diabetes mellitus in Upper Egypt.

BACKGROUND: Diabetes mellitus is a leading cause of morbidity and mortality among children across the world and is responsible for a growing proportion of global healthcare expenditure. However, limited data are available on lung dysfunction in children with diabetes. AIM: The aim of this study was to evaluate the pulmonary function changes in children with type 1 diabetes mellitus (T1DM). METHODS: We studied 60 children with T1DM (mean age 10.5 ± 2.32 years; disease duration 2.45 ± 0.6 years, and 50 healthy control children (mean age 9.9 ± 2.5 years). Spirometry was performed for all individuals to measure forced vital capacity (FVC), forced expiratory volume in 1 second (FEV1), FEV1/FVC ratio, and peak expiratory flow rate (PEFR). Glycemic control was assessed on the basis of glycated hemoglobin (HbA1c), with HbA1c values <8% considered to indicate good glycemic control, and HbA1c values ⩾8% to indicate poor control. RESULTS: There was significant reduction in all spirometeric parameters in diabetic children in comparison with healthy control children. Children with poor glycemic control had significant impairment in lung functions compared with those with good glycemic control. CONCLUSIONS: T1DM in children leads to impairment of lung functions and this impairment increases with poor glycemic control.

Pulse cyclophosphamide therapy in refractory warm autoimmune hemolytic anemia: a new perspective.

Treatment of steroid refractory autoimmune hemolytic anemia (AIHA) is challenging especially with no evidence based consensus guide lines and limited resources. The aim of this study was to evaluate the efficacy of pulse cyclophosphamide therapy in patients with severe refractory warm AIHA. The prospective study was designed to evaluate the efficacy of pulse cyclophosphamide-1 g/month for four consecutive months-in 17 patients (10 males and 7 females) with severe refractory warm AIHA [13 primary AIHA and 4 (females) secondary to SLE], all studied patients failed to respond to high dose of steroid therapy ± azathioprine ± intravenous immunoglobulin ± oral cyclophosphamide. Mean hemoglobin level, reticulocytic count and direct antiglobulin test were assessed before and after cyclophosphamide treatment every month. After the 4th cycle of cyclophosphamide (82 %, 14 patients) achieved partial response while the remaining (17 %, 3 patients) showed no response, while after 6 months follow up 47 % (8 patients) show complete response, while 53 % (9 patients) showed partial response. The mean hemoglobin levels were significantly increased after the 1st, 2nd, 3rd and 4th months of pulse cyclophosphamide therapy when compared to before treatment (P < 0.01, P < 0.001, P < 0.001 and P < 0.001) respectively, and the mean reticulocyte (%) were significantly decreased after the 2nd, 3rd and 4th months (P < 0.05, P < 0.01 and P < 0.001) respectively. We conclude that pulse cyclophosphamide therapy is well tolerated and induces good response in patients with severe refractory warm AIHA.